Sara Balestracci

Quality of Life in Duchenne Muscular Dystrophy: The Subjective Impact on Children and Parents

Duchenne muscular dystrophy results in a broad spectrum of physical and psychosocial consequences, both to patient and caregivers. This study was aimed to explore health-related quality of life and its possible determinants in Duchenne muscular dystrophy children and in their parents. Caregivers (21 mothers and 6 fathers; mean age, 40.04 years) of 27 Duchenne muscular dystrophy patients (mean age, 11.26 years) completed the validated Children Health Questionnaire-Parent Form 50 and the Family Strain Questionnaire. Children reported significantly lower scores than normative group in 10 of 15 Children Health Questionnaire dimensions. Only the use of wheelchairs (P = .02) and ventilators (P < .001) was significantly associated to lower health-related quality of life in Physical Functioning. On the contrary, Family Strain Questionnaire scores were not influenced by children’s characteristics. Despite the presence of Duchenne muscular dystrophy deeply impairs health-related quality of life, some areas of well-being are present both in children and caregivers.

Does asthma control correlate with quality of life related to upper and lower airways? A real life study

Background:  The goal of asthma therapy is to achieve an optimal level of disease control, but the relationship between asthma control, impact of comorbid rhinitis and health related quality of life (HRQoL) in real life remains unexplored.

Disability in COPD and its relationship to clinical and patient-reported outcomes

Abstract Objective: To assess the presence of disability in chronic obstructive pulmonary disease (COPD) patients and its relationships with disease severity, comorbidities, and patient-reported outcomes. Research design and method: COPD outpatients completed validated questionnaires designed to investigate illness perception, well-being, quality of life, and stress, while physicians collected data concerning disability, dyspnea, and comorbidities (Charlson Index). Results: Of 164 patients, 37.3% exhibited a degree of disability and 67.7 % of them reported the loss of at least one relevant function in daily life (mean 2.34 ± 2.41). Although disability was associated with disease severity (χ2 = 8.292; p < 0.016), disability was present to some degree in all disease stages and in 44.9% of patients with moderate COPD. Barthel Index scores were related to MRC scores (r = 0.529; p < 0.001), GOLD stage (r = 0.223; p < 0.006), and Charlson Index (r = 0.163; p < 0.032). Disabled patients had a lower mean FEV1 value (50.96 ± 20.99 vs. 65.00 ± 23.63; p < 0.001) than self-sufficient patients (p < 0.001). The stepwise regression analysis showed that the MRC score was the most relevant factor in inducing COPD patient disability (F = 56.5; p = 0.001). Compared to self-sufficient patients, disabled patients reported lower levels of well-being and health status, increased levels of distress, and a different illness perception. Conclusions: Disability can be identified in each disease stage, with dyspnea serving as the most relevant inducing factor. Since disability substantially impacts patient perception of and experience with COPD, its presence must be taken into account during disease management. The cross-sectional nature of the study and the characteristics of the sample size represent a limitation in the possibility to generalize the results.

RhinAsthma patient perspective: a short daily asthma and rhinitis QoL assessment.

The present study aimed to develop a short validated patient‐completed questionnaire, the RhinAsthma Patient Perspective (RAPP), to assess the health‐related quality of life (HRQoL) in patients with asthma and comorbid allergic rhinitis in clinical practice.

Chronic obstructive pulmonary disease patient well-being and its relationship with clinical and patient-reported outcomes: a real-life observational study.

Background: Quality of life is an umbrella concept that refers to all aspects of a person’s life, including health status and well-being. While health status measure focuses on the impact of the disease on physical functioning, well-being represents the self-representation of the emotional states related to the disease itself. Objectives: The objective of this study was to evaluate the psychological well-being and its determining factors in a real-life chronic obstructive pulmonary disease (COPD) population and to evaluate if patients with a different well-being differ in illness perception, health status and alexithymia. Methods: Psychological well-being (Psychological General Well-Being Index), health status (SF-36), illness perception (Illness Perception Questionnaire), alexithymia (Toronto Alexithymia Scale), as well as clinical parameters were assessed in COPD out-patients. Results: One hundred and sixty-four patients, with a mean forced expiratory volume in 1 s of 58.5%, were recruited. Forty percent of them had a moderately/severely impaired well-being, not correlated with forced expiratory volume in 1 s and the Charlson index value but significantly with the Medical Research Council score (p = 0.0001) that appeared to be the dominant factor. Patients with impaired well-being showed a different illness perception in terms of correct identification of symptoms, disease consequences, emotional representation and confidence in treatment compared with those having a positive well-being. The latter presented a lower alexithymia prevalence and a better health status. Conclusions: In order to minimize the disease-negative effects on patients’ lives, assessment of well-being and its determining factors, as well as planning specific behavioural, educational and therapeutic interventions seem to be relevant and useful.

The relationship between asthma control and quality-of-life impairment due to chronic cough: a real-life study

BACKGROUND The objective of asthma therapy is to achieve optimal disease control and quality-of-life (QoL) improvements. Good correlation is reported between a composite evaluation of asthma control (the Asthma Control Test [ACT]) and QoL, with emphasis on asthma as a frequent cause of chronic cough and worsening of QoL due to chronic cough. OBJECTIVES To investigate asthma control in real life and to evaluate chronic cough-related impairment of QoL in patients with different degrees of asthma control evaluated by the ACT. METHODS Outpatients awaiting physician visits for asthma evaluation were asked to complete the ACT, the Chronic Cough Impact Questionnaire (CCIQ), and a rhinitis symptom score (Total Symptom Score [TSS] 5). RESULTS A total of 122 patients (mean [SD] age, 44 [15] years; age range, 15-75 years; 61.5% female) were enrolled in the study, of whom 74% reported rhinitis in conjunction with asthma. Overall asthma control was substantially less than optimal (ACT mean score, 14) in 44% of patients. Asthma control correlated to a limited degree with the CCIQ among the total sample. In patients with asthma alone, the correlation between the ACT and the CCIQ was much improved. In patients with comorbid rhinitis, only those with significant symptoms (as determined by the TSS5) reported using rhinitis treatment; among these, no significant correlation between the ACT and the CCIQ was found. CONCLUSIONS Asthma control in real life is less than optimal in substantially few patients. The importance of cough in asthma is highlighted by good correlation between cough and the ACT score in patients without comorbid rhinitis. In patients with comorbid rhinitis, cough appears related more to rhinitis than to asthma control. The combined use of the ACT and the CCIQ may be useful to improve asthma management, especially among patients in whom cough remains a prominent symptom.

Disability in moderate chronic obstructive pulmonary disease: Prevalence, burden and assessment-results from a real-life study

Background: The role of disability and its association with patient-reported outcomes in the nonsevere forms of chronic obstructive pulmonary disease (COPD) has never been explored. Objectives: The aim of this study was to assess, in a cross-sectional real-life study, the prevalence and degree of disability in moderate COPD patients and to assess its association with health status, illness perception, risk of death and well-being. Methods: Moderate COPD outpatients attending scheduled visits were involved in a quantitative research program using a questionnaire-based data collection method. Results: Out of 694 patients, 17.4% were classified as disabled and 47.6% reported the loss of at least one relevant function of daily living. Disabled patients did not differ from nondisabled patients in terms of working status (p = 0.06), smoking habits (p = 0.134) and ongoing treatment (p = 0.823); however, the former showed a significantly higher disease burden as measured by illness perception, health status and well-being. The stepwise regression analysis showed that the modified Medical Research Council (mMRC) score was the most relevant factor related to COPD disability (F = 38.248; p = 0.001). Patient stratification was possible according to the forced expiratory volume in 1 s (FEV1) value and an mMRC score ≥2, which identified disabled patients, whereas the mMRC values were differently associated with the risk of disability. Conclusion: A significant proportion of individuals with moderate COPD reported a limitation of daily life functions, with dyspnea being the most relevant factor inducing disability. Adding the evaluation of patient-reported outcomes to lung function assessment could facilitate the identification of disabled patients.